Clinical aspects of aplastic anemia

Severe aplastic anemia is a disorder characterized by peripheral pancytopenia and marrow hypoplasia. Although its pathophysiology is understood poorly, the majority of patients appear to have some immunologic destruction or suppression of hematopoietic cells. The only curative therapy to date is allogeneic stem cell transplantation, although the success of palliative immunosuppressive therapies has improved over the last two decades. Making the best therapy choice is complex and often requires balancing very divergent toxicity profiles, both acute and long-term.